A Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy Versus Standard Supportive Therapy Alone in Cardiac Amyloidosis Caused by Transthyretin
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|ClinicalTrials.gov Identifier: NCT03481972|
Recruitment Status : Active, not recruiting
First Posted : March 29, 2018
Last Update Posted : May 14, 2020
Cardiac amyloidosis caused by transthyretin either mutated (in ATTRm amyloidosis) or wildtype (in ATTRwt, formerly senile, amyloidosis) is a rare disease but is diagnosed with increasing frequency thanks to the availability of non-invasive scintigraphy-based means. Cardiac ATTR amyloidosis is characterized by progressive heart failure with a median survival of less than 4 years, and there is no standard treatment for this disease. It was proved that the marketed antibiotic doxycycline (Doxy) disrupts amyloid fibrils in vitro and in animal models synergistically with tauroursodeoxycholic acid (TUDCA). Based on these pre-clinical data, a clinical trial of Doxy/TUDCA in ATTR Amyloidosis (NCT01171859) was conducted.
Treatment was well tolerated and was able to prevent progression of cardiac dysfunction.
|Condition or disease||Intervention/treatment||Phase|
|TTR Cardiac Amyloidosis||Drug: Doxycycline and tauroursodeoxycholic acid Drug: Standard of care||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||102 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase III Randomized Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy Versus Standard Supportive Therapy Alone in Cardiac Amyloidosis Caused by Transthyretin|
|Actual Study Start Date :||April 11, 2018|
|Estimated Primary Completion Date :||April 2021|
|Estimated Study Completion Date :||April 2021|
doxycicline (100 mg / BID)
tauroursodeoxycholic acid (250 mg / TID)
Drug: Doxycycline and tauroursodeoxycholic acid
doxycicline and tauroursodeoxycholic acid
Other Name: Standard of care therpay
Active Comparator: Standard of care
Standard of care therapies.
Drug: Standard of care
Standard of care
- evaluate the efficacy of Doxy/TUDCA in patients with cardiac ATTR amyloidosis, comparing survival free at 18 months of patients receiving Doxy/TUDCA + standard supportive therapy with that of patients in standard supportive therapy alone. [ Time Frame: 18 MONTHS ]Doxy/TUDCA efficacy
- Compare overall survival at 18 and 30 months of patients receiving Doxy/TUDCA in addition to standard supportive therapy with that of patients receiving standard supportive therapy alone. [ Time Frame: 18 and 30 months ]Survival
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03481972
|Fondazione IRCCS Policlinico San Matteo|
|Pavia, Italy, 27100|